Abstract:
:Large electronic health care databases can be used to evaluate the effectiveness of regulatory action. Designing and interpreting such studies can be challenging. We demonstrate using case studies on modafinil, benzodiazepines, dosulepin, and piroxicam that measuring the impact of regulatory action is possible and can be beneficial, particularly in determining the need for further action or communication. For each case study, we discuss difficulties that may arise when measuring the effectiveness of regulatory action, which include but are not limited to: the choice of denominator, changes in the patient population, the availability and selection of comparators, and the fact that the regulatory voice may be but one voice among many. The evaluation of regulatory action is possible, but not without its challenges, and a routine, one-size-fits all approach is unlikely to be possible.
journal_name
Ther Innov Regul Scijournal_title
Therapeutic innovation & regulatory scienceauthors
Thomson A,Meeraus WH,Wong J,Suvarna Rdoi
10.1177/2168479015570333subject
Has Abstractpub_date
2015-07-01 00:00:00pages
473-482issue
4eissn
2168-4790issn
2168-4804journal_volume
49pub_type
杂志文章abstract:BACKGROUND:Safety data are continuously evaluated throughout the life cycle of a medical product to accurately assess and characterize the risks associated with the product. The knowledge about a medical product's safety profile continually evolves as safety data accumulate. METHODS:This paper discusses data sources a...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章,评审
doi:10.1177/2168479017741112
更新日期:2018-03-01 00:00:00
abstract:OBJECTIVE:To identify, evaluate, and characterize the variety, quality, and intent of the health economics and outcomes research studies being conducted in SAARC (South Asian Association for Regional Cooperation) nations. METHODS:Studies published in English language between 1990 and 2015 were retrieved from Medline d...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章,评审
doi:10.1177/2168479017731583
更新日期:2018-05-01 00:00:00
abstract:BACKGROUND:A new incentive program, "Premium Rewards for the Promotion of Innovative Drug Discovery and Resolution of Off-Label Use, etc," was introduced in Japan in 2010 and subsequently applied in 2010, 2012, and 2014. In our previous study, we reported statistical evidence regarding the attributes of drugs for which...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479015600205
更新日期:2016-01-01 00:00:00
abstract::When registering food supplements in Russia, restrictions on the labeling and consumer leaflet contents are tightly controlled by the regulatory agencies. Thus, misleading advertising is the most difficult problem with the turnover of these products if information in the commercials does not correspond to the informat...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章,评审
doi:10.1007/s43441-019-00043-z
更新日期:2020-01-01 00:00:00
abstract:BACKGROUND:(1) A growing number of pharmaceutical and biotechnology organizations are engaging patients, their support networks, and clinical trial site staff at various touchpoints along the clinical research development spectrum to solicit feedback on how to reduce the burden of clinical trial participation and admin...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479019849875
更新日期:2019-05-22 00:00:00
abstract::TransCelerate BioPharma Inc developed a methodology based on the notion that shifting monitoring processes from an excessive concentration on source data verification to comprehensive risk-driven monitoring will increase efficiencies and enhance patient safety and data integrity while maintaining adherence to good cli...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479014546336
更新日期:2014-09-01 00:00:00
abstract:BACKGROUND:The number of diabetes patients is steadily increasing worldwide. Consequently, the social burden of diabetes is huge, requiring urgent countermeasures. We performed an intensive survey of antidiabetic drugs approved in Japan, the United States, and the European Union. METHODS:Information about approved ant...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
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abstract:BACKGROUND:The influence of patient advocates on FDA regulatory decision making has increased. Despite enhanced engagement with FDA, there remain challenges to achieving the regulatory goals of patients within FDA's regulatory framework. Gaps exist between patient advocates' knowledge of the agency's processes and FDA'...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
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abstract:BACKGROUND:Approval of regulated medical products in the USA is based upon a rigorous review of the benefits and risks as performed by the US Food and Drug Administration (FDA) staff of scientists and is summarized in a descriptive and qualitative format called the FDA's Benefit-Risk Framework (BRF). This present metho...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-020-00197-1
更新日期:2021-01-01 00:00:00
abstract::After nearly a decade of discussion, analysis, and development, the Medicines Adaptive Pathways to Patients (MAPPs) initiative is beginning to see acceptance from regulators, industry, patients, and payers, with the first live pilot project initiated under the guidance of the European Medicines Agency in 2014. Althoug...
journal_title:Therapeutic innovation & regulatory science
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更新日期:2016-05-01 00:00:00
abstract:BACKGROUND:Prescription drug labeling is an authoritative source of information that guides the safe and effective use of approved medications. In many instances, however, labeling may fail to be updated as new information about drug efficacy emerges in the postmarket setting. When labeling becomes outdated, it loses i...
journal_title:Therapeutic innovation & regulatory science
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doi:10.1177/2168479018759662
更新日期:2018-11-01 00:00:00
abstract::Adaptive designs are increasingly used in clinical trials. The Drug Information Association's Adaptive Design Scientific Working Group (ADSWG) works to foster collaboration among regulatory agencies, academia, and pharmaceutical and biotech companies to further the science of adaptive clinical development. The ADSWG S...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479014522468
更新日期:2014-07-01 00:00:00
abstract::The product pipeline for diseases that disproportionately affect the developing world has considerably expanded over the last decade. Indeed, there are about 134 products for these diseases in the pipeline, including vaccines, drugs, diagnostics, microbicides, and vector control tools, and dozens of these products are...
journal_title:Therapeutic innovation & regulatory science
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更新日期:2013-05-01 00:00:00
abstract::A growing number of organizations-including pharmaceutical and biotechnology companies, foundations and associations-are routinely implementing patient advisory boards (PAB) given their high reported value for minimal relative investment. Organizations are typically implementing PABs to solicit patient voices and pers...
journal_title:Therapeutic innovation & regulatory science
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更新日期:2018-07-01 00:00:00
abstract:BACKGROUND:The pace of innovation, the creative engine of the pharmaceutical industry, has been variably described as stagnant, stable, or accelerating depending on the metric used for assessment or the quality of evidence. If the predominant perception holds that the speed of innovation is sluggish, pressure for chang...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-020-00210-7
更新日期:2020-08-31 00:00:00
abstract:BACKGROUND:According to 21 Code of Federal Regulation (CFR) Part 211, Over-the-Counter (OTC) drug manufacturers must establish and follow current good manufacturing practices (cGMP) to produce quality products while meeting regulatory standards. The warning letters issued by the U.S. Food and Drug Administration (FDA) ...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-020-00231-2
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abstract::Risk-based monitoring (RBM) has disrupted the clinical trial industry, challenging conventional monitoring norms, business processes, and organizational structures. Endorsed by regulators and leading industry forums, and further driven by escalating drug development costs and enabling technology shifts making data ava...
journal_title:Therapeutic innovation & regulatory science
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更新日期:2019-03-01 00:00:00
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journal_title:Therapeutic innovation & regulatory science
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doi:10.1007/s43441-020-00161-z
更新日期:2020-11-01 00:00:00
abstract:BACKGROUND:Timely access to new medicines may be addressed through strengthening of registration efficiencies and timelines by establishing and refining value-added registration processes, resources, and systems. The aims of this study were to evaluate the timelines of the milestones of the South African review process...
journal_title:Therapeutic innovation & regulatory science
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doi:10.1007/s43441-019-00013-5
更新日期:2020-07-01 00:00:00
abstract::There is a long history of discussions about what is a minimal clinically important difference (MCID) and how this term applies to clinical research. This paper deals with a practical framework for MCID and its applicability to clinical trials. A literature review on the topic confirmed the fundamental role of MCID fo...
journal_title:Therapeutic innovation & regulatory science
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abstract::The DIA Study Endpoints Community Working Group on Mobile Sensor Technology (MST) series addresses considerations that may be useful for selecting MST for use in a clinical trial. This article describes considerations regarding the selection of MST for clinical trials including expectations around technology specifica...
journal_title:Therapeutic innovation & regulatory science
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journal_title:Therapeutic innovation & regulatory science
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journal_title:Therapeutic innovation & regulatory science
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doi:10.1177/2168479014558273
更新日期:2015-03-01 00:00:00
abstract::When does a single positive adequate and well-controlled study of a new drug meet the statutory requirement to demonstrate substantial evidence of effectiveness? The answer to this question, particularly with respect to new molecular entities, has been of considerable debate since 1962 when the requirement that new dr...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-019-00103-4
更新日期:2020-05-01 00:00:00
abstract::Recently, it is becoming increasingly difficult to develop innovative drugs. Thus, the role of regulatory science research in drug development and postmarketing settings has become more important. In this article, the authors discuss the roles of regulatory science research at the Pharmaceuticals and Medical Devices A...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479012469950
更新日期:2013-01-01 00:00:00
abstract:BACKGROUND:The US Food and Drug Administration (FDA) put out a call for comments on new draft guidance for industry "Comparative Analyses and Related Comparative Use Human Factors Studies for a Drug-Device Combination Product Submitted in an ANDA." This call for comments elicited 7 submissions from various organization...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章,评审
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更新日期:2019-03-01 00:00:00
abstract::The authors consider a statistically valid method that has little concern caused by down weighting in stage 2 or slight loss in efficiency and possibly improves power performance for a trial design that allows for adaptation of statistical information. In addition to the need for a priori sound data-based planning tha...
journal_title:Therapeutic innovation & regulatory science
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更新日期:2014-01-01 00:00:00
abstract::This study aimed to determine the potentially severe chemical properties of drugs that can cause adverse drug reactions (ADRs) such as erythema multiforme (EM), Stevens-Johnson syndrome (SJS), and toxic epidermal necrolysis (TEN) by using a data mining method. The study data were extracted from the Adverse Event Repor...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/0092861512460759
更新日期:2013-03-01 00:00:00
abstract::The volume and diversity of data collected to support each clinical study has increased dramatically in response to the rising scope and complexity of global drug development programs. The Tufts Center for the Study of Drug Development conducted an online survey of 257 unique global companies-77% drug development spon...
journal_title:Therapeutic innovation & regulatory science
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更新日期:2019-01-01 00:00:00
abstract::Using a measure of agreement that does not distinguish the "positive" outcome from the "negative" outcome can be sometimes misleading in assessing resemblance. To alleviate this concern, some new indices, including the "positive" and "negative" conditional synchrony measures (CSM) (or the conditional discordant measur...
journal_title:Therapeutic innovation & regulatory science
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